Genetic medicine can leave people with rare mutations behind. But there’s new hope
By Laura Ungar,
Associated Press
| 04. 26. 2025
Emily Kramer-Golinkoff can’t get enough oxygen with each breath. Advanced cystic fibrosis makes even simple things like walking or showering arduous and exhausting.
She has the most common fatal genetic disease in the U.S., which afflicts 40,000 Americans. But her case is caused by a rare genetic mutation, so medications that work for 90% of people with cystic fibrosis won’t help her.
The same dynamic plays out in other genetic conditions. Stunning advances in genetic science have revealed the subtle, insidious culprits behind these brutal diseases and have started paving the way for treatments. But patients with these exceedingly rare mutations have fewer options and poorer prospects than those with more typical forms of these diseases — and many are now pinning hopes on experimental gene therapies.
“We feel such pure joy for our friends who have been lifted from this sinking ship,” said Kramer-Golinkoff, 40. “But we just feel so eager and desperate to join them. It’s really hard to be in this minority of people left behind.”
It’s not just science that is working against these patients...
Related Articles
GeneWatch UK has prepared a briefing on the genetic modification of nature for the International Union for Conservation of Nature (IUCN) Congress in October 2025
The upcoming Congress claims to be “where the world comes together to set priorities and drive conservation and sustainable development action.” A major concern for those on the outside is that the Congress may advance plans to develop and encourage the use of synthetic biology in nature conservation. This could at first glance sound like...
By Aaron Ginn, The Washington Post | 09.12.2025
Earlier this year, I had dinner in D.C. with Jensen Huang, the president and chief executive of Nvidia. At one point, he said something that struck me: “Why is everyone here so negative?”
He wasn’t referring to the economy...
By Roni Caryn Rabin, The New York Times | 08.25.2025
Scientists have dreamed for centuries about using animal organs to treat ailing humans. In recent years, those efforts have begun to bear fruit: Researchers have begun transplanting the hearts and kidneys of genetically modified pigs into patients, with varying degrees...
The Center for Genetics and Society is delighted to recommend the current edition of GMWatch Review – Number 589. UK-based GMWatch, a long-standing ally, was founded in 1998 by Jonathan Matthews as an independent organization seeking to counter the enormous corporate political power and propaganda of the GMO industry and its supporters. Matthews and Claire Robinson are its directors and managing editors.
CGS works to ensure that social justice, equity, human rights, and democratic governance are front...